AstraZeneca’s Koselugo Recommended For EU Approval in Adult NF1 With Plexiform Neurofibromas

AstraZeneca announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending approval of Koselugo (selumetinib) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adults with neurofibromatosis type 1 (NF1).

Prof. Pierre Wolkenstein, MD, PhD, Head of the Department of Dermatology at Henri Mondor Hospital, APHP, Paris East University (UPEC), and National Coordinating Investigator of the KOMET trial in Europe, said, “For adults with NF1, tumour growth doesn’t stop at childhood and can rapidly progress or develop into adulthood, impacting physical, emotional and social well-being. The positive recommendation by the CHMP for Koselugo in adults underscores the urgent need for additional targeted treatments for this patient population. When approved, Koselugo could offer a treatment option for adult patients and continuity of care across age groups, supported by established clinical experience and practice among doctors managing this lifelong condition.”

The recommendation is based on results from the phase III KOMET trial, the largest and only placebo-controlled global study in this patient population. Data presented at the 2025 ASCO Annual Meeting and published in The Lancet showed Koselugo delivered a statistically significant objective response rate (ORR) of 20% versus 5% with placebo (p=0.01) at cycle 16.

NF1 is a rare genetic disorder that often begins in childhood but persists throughout life, impacting multiple organ systems. Up to 50% of people with NF1 may develop plexiform neurofibromas, non-malignant tumors that can grow extensively, causing pain, disfigurement, functional impairment, and muscle weakness. Currently, treatment options are limited, and inoperable PN remain a major unmet need.

Marc Dunoyer, Chief Executive Officer, Alexion, said, “The CHMP positive opinion of Koselugo in adults with NF1 PN builds on more than a decade of global clinical and real-world experience, reflecting the unmatched body of evidence supporting the safety profile and efficacy of Koselugo. As demonstrated by the results from KOMET, the largest and only placebo-controlled, double-blind global Phase III trial in an adult population, we continue to advance the pioneering legacy of Koselugo, which set the treatment standard in NF1 PN, to reach even more people worldwide.”

Koselugo’s safety profile in adults was consistent with its established use in pediatric patients with NF1. The medicine has already been approved in Japan and other markets for this indication, with further regulatory reviews underway globally.  If approved, Koselugo would be the first targeted therapy for adult NF1 patients with symptomatic, inoperable PN in the EU, offering a much-needed treatment option.